Neurogene followed up a premarket setback to its Rett syndrome program Monday with a postmarket confirmation that development of another gene therapy has hit a wall.
The postmarket update covered NGN-101, a gene therapy Neurogene was developing as a treatment for CLN5 Batten disease, and repeated what the biotech disclosed last week. Three months ago, the biotech said it planned to meet with the FDA and try to align with the agency on clinical requirements to support a streamlined registration pathway. Neurogene framed alignment on a streamlined pathway as critical for continued investment in the program.
Talks with the FDA haven’t gone according to plan. Seeking a streamlined pathway, Neurogene submitted a Regenerative Medicine Advanced Therapy (RMAT) application to the FDA. The biotech said the filing met the standard of preliminary clinical evidence required to obtain an RMAT designation.
However, the FDA denied the application. Neurogene responded by dropping plans to advance NGN-101. The decision reflects the rarity of CLN5 Batten disease, which led the biotech to conclude it would need to align with the FDA on a streamlined registrational pathway to support further investment in the asset. Neurogene said in a release that it is now evaluating options for the program.
The biotech licensed the CLN5 gene therapy program from the University of North Carolina at Chapel Hill (UNC) back in 2019. The deal was small, with Neurogene agreeing to pay UNC up to $1.7 million in sales-related milestones and making five-figure payments tied to the progress of the program. UNC received $15,000 when the candidate secured orphan drug designation and $30,000 when phase 1 dosing began.
The UNC deal led to Neurogene becoming the first company to test a gene therapy in people with CLN5 Batten disease. The biotech recently completed enrolling the six subjects in its phase 1/2 dose-escalation trial. Other organizations are working on treatments for CLN2 and CLN3 disease, but NGN-101 is the only CLN5 therapy listed on ClinicalTrials.gov.
Neurogene’s stock plummeted after it disclosed the setback to the Rett gene therapy, closing down more than 40% at below $20.
Editor’s Note: This article was updated at 11 am ET on Nov. 19 to say Neurogene previously disclosed the CLN5 update.